Search
Research
Rhinovirus Infection Is Associated With Airway Epithelial Cell Necrosis and Inflammation via Interleukin-1 in Young Children With Cystic FibrosisThis study found rhinovirus infection drives necrotic cell death in cystic fibrosis airway epithelial cells
Research
Airway macrophages display decreased expression of receptors mediating and regulating scavenging in early cystic fibrosis lung diseaseCystic fibrosis (CF) airway disease is characterized by chronic inflammation, featuring neutrophil influx to the lumen. Airway macrophages (AMs) can promote both inflammation and resolution, and are thus critical to maintaining and restoring homeostasis. CF AM functions, specifically scavenging activity and resolution of inflammation, have been shown to be impaired, yet underlying processes remain unknown.
Research
Prevalence of tracheobronchomalacia is higher than previously reported in children with cystic fibrosisTracheobronchomalacia (TBM) is estimated to be present in 1 in 2100 children. Previous reports suggest the prevalence is higher in children with cystic fibrosis (CF). This has clinical implications with potential to influence airway clearance and lung health.
Research
BEAT CF pulmonary exacerbations core protocol for evaluating the management of pulmonary exacerbations in people with cystic fibrosisCystic fibrosis (CF) is a rare, inherited, life-limiting condition predominantly affecting the lungs, for which there is no cure. The disease is characterized by recurrent pulmonary exacerbations (PEx), which are thought to drive progressive lung damage. Management of these episodes is complex and generally involves multiple interventions targeting different aspects of disease. The emergence of innovative trials and use of Bayesian statistical methods has created renewed opportunities for studying heterogeneous populations in rare diseases.
Research
Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse ModelSeverity and disease progression in people with Cystic Fibrosis is typically dependent on their genotype. One potential therapeutic strategy for people with specific mutations is exon skipping with antisense oligonucleotides. CFTR exon 9 is an in-frame exon and hence the exclusion of this exon would excise only 31 amino acids but not alter the reading frame of the remaining mRNA.
Research
The effect of azithromycin on structural lung disease in infants with cystic fibrosis (COMBAT CF): a phase 3, randomised, double-blind, placebo-controlled clinical trialStructural lung disease and neutrophil-dominated airway inflammation is present from 3 months of age in children diagnosed with cystic fibrosis after newborn screening. We hypothesised that azithromycin, given three times weekly to infants with cystic fibrosis from diagnosis until age 36 months, would reduce the extent of structural lung disease as captured on chest CT scans.
Research
Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: A systematic reviewThere is no consensus about which outcomes should be evaluated in studies of pulmonary exacerbations in people with cystic fibrosis (CF). Outcomes used for evaluation should be meaningful; that is, they should capture how people feel, function or survive and be acknowledged as important to people with CF, or should be reliable surrogates of those outcomes. We aimed to summarise the outcomes and corresponding endpoints which have been reported in studies of pulmonary exacerbations, and to identify those which are most likely to be meaningful.
Research
Structural determinants of long term functional outcomes in young children with cystic fibrosisChest CT identifies children at an early age who have adverse long-term outcomes
Research
Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized MedicineIn this Review, we consolidate the latest updates in the development of primary epithelial cellular model systems relevant for cystic fibrosis
Research
Trial refresh: A case for an adaptive platform trial for pulmonary exacerbations of cystic fibrosisThis review will discuss the necessary steps required for a Bayesian adaptive platform trial to optimize treatment of pulmonary exacerbations of cystic fibrosis